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11 Cards in this Set

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Reproduction System- Stem Cell Therapy by Thatcher
Reproduction System- Stem Cell Therapy by Thatcher
Recognize strategies for treating genetic disorders, and list examples of diseases treated by different strategies
in some packet.
Discuss principles for providing appropriate genetic counceling
1.consult patient
2. perform risk assessments (eg., family history)
3. testing
4. treatment
5. consult patient (informed consent, confidentiality)
*non-directive- don't tell what to do, just give the information.
Recognize what GINA does and does not cover
GINA (Genetic Information Non-Discrimination Act)

 Prohibits health insurers from using genetic information in determining eligibility or setting premiums

Prohibits employers from purchasing a person’s genetic information, or using genetic information in decisions regarding hiring, firing, work assignments, etc.

Prohibits health insurers or employers from requesting or requiring a person or family member to undergo a genetic test


*can't use genetic information against you. not required to take genetics test.
*does NOT cover life insurance, medical underwriting, apply to military personnel, prevent physicians from ordering genetic tests, mandate coverage for specific genetic testing or tx.
List advantages and limitation of different strategies for gene therapy.
Germ Line Transformation: replace a mutant gene with a wild type allele in the germ line (added to blast cells, eggs or sperm); can eliminate the defective gene; Microinjection, Homologous recombination; Experimental tool, e.g. gene knockout mice

Somatic Transformation: deliver a wild type gene to somatic cells of an adult or child, without eliminating the mutant allele; All current trials

In vivo- transformation of cells within the body
Ex vivo- transformation of extracted cells, which are then reimplanted back into the body.

Direct:
Gene Replacement: add wild type gene to cells with mutant genes
RNAi (inhibits): inhibits expression of specific gene

Indirect (most target cancerous cells):
Suicide genes: toxic genes, expression often activated by prodrug
Immune Response: antigenic genes, transformed cells cleared
List challenges for developing effective gene therapies
-Transformation Level (non-dividing cells difficult)
-Expression Level
-Regulation (correct spacial, temporal, stimuli responsive expression)
-Targeting Specific Organ or Cell Type
-Transformation Stability (biggest problem)
--Chromosomal Integration
--Immunogenicity (transformed cells cleared, often increases with repeated treatments)

Multifactoral traits: transforming patients with multiple transgenes is impractical, but it may be reasonable to treat specific diseases by transforming patients with one or a few critical genes)

Gene size: large genes more difficult.
Discuss the advantages and disadvantages of using viral vectors for gene therapy, and the advantages and disadvantages specific to the three major viral vectors.
Naturally Infect Host Cells; Cell Type Specificity; Genetic Engineering
(Replication defective; Restricts gene size)

Chromosomal Integration (Retroviruses):
Transformation stable; Transformation propagated in daughter cells; Insertional mutagenesis; Chromosomal integration (stable transformation transformation propagated in daughters; insertional mutagenesis); require a dividing cell, limited transcript size

Extrachromosomal Replication (Adenoviruses): Transient transformation; No risk of insertional mutagenesis; DNA viruses; wide range of targets; don't require cell division; accommodate larger genes (8-30kb); transient transformation; immunogenic

Adeno-Associated Viruses: require adenovirus co-infection; target brain and muscle; small capacity (about 5kb)
Describe methods for hematopoietic transplantation stem cell therapy
Viral Infection- can genetically engineer them
Lipofection - package vector in membranous vesicles
Condensed DNA Particles - package vector in protein coat
Molecular Conjugates - bind DNA to ligand, usually protein
Naked DNA Injection - inject DNA directly into solid structures (e.g. tumors, muscles)
Electroporation - run an electric current through cells
ex vivo or dermal
Gene Gun - shoot gold particles coated with DNA into
cells with a high-pressure helium stream . ex vivo or dermal
List challenges for developing effective stem cell therapies
Seed Threshold- stem cell quantity must be high enough for graft to take

Cell culture expansion- premature differentiation needs to be controlled.

Controlling differentiation- growth factors to prevent premature differentiation and control differentiation.

Immunogenic rejection- body recognizes as foreign, the stem cells are eliminated as you put them in.

Autologous- patient's own cells; no rejection.

Allogeneic- cell from a matched donor.

Infection-

Development of secondary cancer-
Describe the advantages and disadvantages of adult and embryonic stem cell therapies
Adult stem cells: mesenchymal; undifferentiated (more differentiated than embryonic stem cells)

Embryonic stem cells: isolated from inner cell mass of a blastocyst; pluripotent (less differentiated than adult stem cells); ethical issues
Name some dangers with gene therapy
Immune Responses: when immunocompromised, systemic immunologic reaction ex anaphylactic shock

Horizontal Transformation: from transformed cells to another cell
type that should not be transformed, e.g. from somatic to germ line cells

Pathogenic Vector Development: inadvertently creating a new pathogen e.g. replication competent viral reversion due to mutations

Insertional Mutagenesis: (XSCIDS case) ; gene inserting into another gene. the virus may carry regulatory sequences with it.